FDA Grants Orphan Drug Designation to DOC1021 for Malignant Glioma


The completion of the phase 1 trial is a crucial step toward making DOC1021 available to all patients with glioblastoma multiforme.

  • In the phase 1 trial (NCT04552886) evaluating DOC102 for glioblastoma multiforme (GBM), enrollment has been successfully completed.

  • The FDA also granted DOC1021 an orphan drug designation for malignant glioma, including GBM, signifying its potential broader applicability to other oncological indications.

  • DOC1021 has demonstrated strong safety and efficacy signals, with no attributable serious adverse events observed.

Enrollment in a phase 1 trial evaluating DOC102 for the treatment of GBM has been completed, and the FDA has granted DOC1021 orphan drug designation for malignant glioma, including GBM, according to Diakonos Oncology Corporation.1

“Receiving the orphan drug designation from the FDA is a valuable addition to our existing fast track designation as it indicates that the cell therapy may be more broadly applicable to other oncological indications,” said Ian Bellayr, PhD, chief regulatory officer of Diakonos, in a press release. “As we continue to accumulate more safety and efficacy data with time, Diakonos will continue to leverage other FDA programs to expedite development.”

DOC1021 is a unique vaccine which is made with dendritic cells from a patient as well as a sample of their tumor. With the use of dendritic cells, the complete cancer antigen profile is able to be targeted in a patient without any genetic modification of their immune cells. Further, the vaccines employ a natural immune response that can target and eliminate cancer cells as if they were virally infected.

The first patient was enrolled in October 2021 into the non-randomized, single-arm, phase 1 trial and to date, 16 patients have been given DOC1021 across 4 dose levels. Among these patients, DOC1021 has been safe and well tolerated with no attributable serious adverse events observed thus far. Of the 16, 13 patients are alive and most remain progression-free.

glioblastoma multiforme: © peterschreiber.media - stock.adobe.com

glioblastoma multiforme: © peterschreiber.media - stock.adobe.com

"Completing the phase 1 trial with both strong safety and efficacy signals was a critical step towards providing DOC1021 to all GBM patients,” said Mike Wicks, chief executive officer of Diakonos, in a press release. “Our focus now shifts towards manufacturing optimization underway with Cellipont Bioservices and enrolling additional sites for the upcoming phase 2 trial.”

In the phase 1 trial of DOC1021 for GBM, investigators are evaluating the safety and feasibility of this dendritic cell vaccinein adult patients with GBM after undergoing neurosurgical tumor resectionand in whom a neuropathological diagnosis has been established. A 3+3 design is being utilized in this study for 3 steps of dose-escalation with mandatory safety monitoring.2

Enrollment in the study is open to patients aged 18 years and older with GBM that is potentially resectable and who are deemed to be good candidates for postoperative adjuvant chemotherapy and radiation therapy. Patients must be able to adhere to the bi-weekly injections of treatment regimen; have adequate kidney, liver, bone marrow function, and immune function; an ECOG performance status of ≤ 2; and be presented at the tumor board for review and consensus of a multidisciplinary group to proceed with enrollment.

Female patients of reproductive potential must use highly effective contraception for at least 1 month before screening and must agree to use such a method during study participation and for an additional 12 weeks following discontinuations of last vaccination. They must also have a negative serum pregnancy test prior to first treatment. Males of reproductive potential must also agree to use condoms or other methods to ensure effective contraception during the course of study participation, as well as for an additional 12 weeks following discontinuations of last vaccination.

In addition to DOC1021, 2 other vaccines are undergoing clinical development for the treatment of patients with pancreatic cancer (NCT04157127) and angiosarcoma (NCT05799612).1

Diakonos Oncology completes phase 1 glioblastoma trial recruitment; receives FDA orphan drug designation. News release. Diakonos Oncology Corporation. January 2, 2024. Accessed January 3, 2024. http://tinyurl.com/4jmdnjdn
Dendritic cell vaccination with standard postoperative chemoradiation for the treatment of adult glioblastoma. ClinicalTrials.gov. Updated June 28, 2022. Accessed January 3, 2024. https://clinicaltrials.gov/study/NCT04552886
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